Gene therapy for ADA-SCID: Defining the factors for successful outcome

Abstract

In this issue of Blood, Carbonaro et al1 and Candotti et al2 demonstrate, in the clinical and nonclinical settings, the absolute need for cytoreductive conditioning in successful treatment of adenosine deaminase - deficient severe combined immune deficiency (ADA-SCID) by gene therapy. Intriguingly and in contrast to previously held paradigms, Carbonaro et al suggest that the continued use of enzyme replacement therapy (ERT) after gene therapy may improve immune reconstitution.